CRISPR/Cas9 technology in the modeling of and evaluation of possible treatments for Niemann-Pick C

Niemann–Pick disease type C (NPC) is a rare neurodegenerative condition resulted from mutations in NPC1 and NPC2 genes. This cellular lipid transferring disorder mainly involves endocytosed cholesterol trafficking. The accumulation of cholesterol and glycolipids in late endosomes and lysosomes res...

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Veröffentlicht in:	Molecular biology reports 2024-12, Vol.51 (1), p.828, Article 828
Hauptverfasser:	Reyhani-Ardabili, Mehran, Fathi, Mohadeseh, Ghafouri-Fard, Soudeh
Format:	Artikel
Sprache:	eng
Schlagworte:	Animal Anatomy Animal Biochemistry Animals Apoptosis Ataxia Biomedical and Life Sciences Brain diseases Cell adhesion & migration Cholesterol Cholesterol - metabolism Convulsions & seizures CRISPR CRISPR-Cas Systems - genetics Disease Models, Animal Endosomes Enzymes Gene Editing - methods Gene therapy Genetic disorders Genetic engineering Genetic Therapy - methods Genome editing Genomes Glycolipids Histology Humans Life Sciences Lysosomes Metabolic disorders Molecular biology Morphology Mutation Neurodegeneration Niemann-Pick C1 Protein Niemann-Pick disease Niemann-Pick Disease, Type C - genetics Niemann-Pick Disease, Type C - metabolism Niemann-Pick Disease, Type C - therapy Npc1 protein Protein transport Proteins Review
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